Aislinn Antrim, Associate Editor

The FDA has approved evolocumab (Repatha; Amgen) for the treatment of pediatric patients 10 years of age and older with heterozygous familial hypercholesterolemia (HeFH) to reduce low-density lipoprotein cholesterol (LDL-C).

HeFH is a genetic condition affecting 1 in 250 people worldwide, according to an Amgen press release. It is estimated that 1 million people in the United States have familial hypercholesterolemia (FH), although fewer than 10% are diagnosed, according to the press release. It is characterized by high LDL-C levels starting at birth, which can accelerate the development of atherosclerotic cardiovascular disease. This can lead to an increased risk of cardiovascular events at an early age, including heart attack and other vascular conditions.

According to the press release, children with FH can have a normal weight, a good diet, and adequate exercise, and can still have LDL-C. The drug is approved as an adjunct to diet and other LDL-C-lowering therapies.

“The approval of Repatha for pediatric patients with FH represents a much-needed adjunct treatment option for these children with genetically high cholesterol who are unable to manage their high LDL-C with other lipid-lowering agents alone,” said David M. Reese, MD, executive vice president of research and development at Amgen, in the press release.

The approval is based on data from the HAUSER-RCT phase 3b study, which evaluated the safety and efficacy of evolocumab in pediatric patients between 10 and 17 years of age who were diagnosed with HeFH. According to the press release, 53 patients received a placebo and 104 patients received 420 mg of evolocumab.

Patients were required to be on a low-fat diet and must have been receiving optimized background lipid-lowering therapy. According to the press release, monthly treatment with evolocumab reduced LDL-C by a mean of 38% from baseline compared to placebo, which showed that the study met its primary endpoint.

LDL-C reductions were also observed by the first post-baseline assessment at the week 12 time point and were maintained throughout the trial. Patients who received evolocumab had improved secondary lipid parameters from baseline compared to placebo, including a 35% reduction in non-high-density lipoprotein cholesterol (non-HDL-C), a 27% reduction in total cholesterol, and a 32% reduction in apolipoprotein B at week 24.

No new safety risks were identified in the trial. The most common treatment-emergent adverse events included nasopharyngitis, headache, oropharyngeal pain, influenza, and upper respiratory tract infection, according to the press release.

“As pediatric FH is an under-recognized condition that can lead to premature coronary artery disease, it’s critically important to have additional treatments that can significantly lower cholesterol,” said Katherine Wilemon, founder and chief executive officer at The FH Foundation, in the press release.

FDA Approves Repatha (evolocumab) In Pediatric Patients Age 10 And Older With Heterozygous Familial Hypercholesterolemia. News release. Amgen; September 24, 2021. Accessed September 27, 2021. 

https://www.amgen.com/newsroom/press-releases/2021/09/fda-approves-repatha-evolocumab-in-pediatric-patients-age-10-and-older-with-heterozygous-familial-hypercholesterolemia

Articles

Monthly treatment with evolocumab reduced LDL-C by a mean of 38% from baseline compared to placebo among patients ages 10 to 17 with heterozygous familial hypercholesterolemia.

FDA Approves Evolocumab for Patients Ages 10 and Older With Heterozygous Familial Hypercholesterolemia

The results of a new report from the Association for Accessible Medicines showed that generic prescriptions are less available to patients with Medicare Part D plans than to those with commercial insurance plans.

Generic medications are an important option to improve health care access and reduce medical costs for patients, especially older adults who often have chronic conditions or take multiple medications.

Generics saved the national health care system $313 billion in 2019 alone and close to $2.2 trillion over the part decade, according to the report.

In 2020, 72 first generics were approved, increasing market competition and helping lower costs for many patients, according to the report.

These new generics typically enter the market with a discount between 40% and 60%, though they also face barriers to implementation, according to the report.

Earlier research results suggested that generic drugs approved by the FDA between 2016 and 2018 had significant delays in Medicare Part D formulary coverage compared with commercial insurance plans.

Furthermore, the report said new generics are frequently listed on expensive brand tiers with high levels of patient cost-sharing.

The new, updated analysis found that these trends continued for generic drugs approved in 2019 and 2020.

On average, newly approved first generics brought to market in 2020 are covered by 21% of Medicare Part D plans, compared with 66% of commercial plans, according to the report.

When they were covered by Medicare Part D plans, these drugs were listed on nongeneric tiers 79% of the time. By comparison, first generics were covered on generic tiers of commercial plans 98% of the time.

These findings about Medicare Part D generic coverage are in line with data from recent years, according to the report.

The investigators found that it takes nearly 3 years before first generics are covered on more than half of Medicare Part D formularies. Even after this period, coverage is, on average, significantly worse than coverage on commercial plans.

"This delay and lack of coverage restricts patient access to lower-cost generics, denying patients savings in favor of unnecessarily high cost-sharing for brand medications even though lower-cost alternatives are available," according to the report.

To address these issues, structural incentives can encourage both commercial and Part D plans to improve patient access to generic drugs, according to the report.

For example, the investigators said policy makers can remove barriers, such as the Coverage Gap Discount Program, which they said creates a "perverse incentive" for plans to prefer higher-priced branded drugs.

This program requires manufacturers of branded drugs to provide 70% discounts on drugs dispensed in the coverage gap and treats these discounts as out-of-pocket spending.

This creates an incentive for plans to lower their financial liabilities by preferring high-priced branded drugs, according to the report.

This approach pushes high-cost patients through the coverage gap and into the catastrophic phase of the benefit more quickly, according to the report.

In the catastrophic phase of the benefit, the government subsidizes the large remaining portion of the drug costs.

"This is a powerful incentive for plans to advantage higher-cost brand drugs over generics, even when those generics cost less and even if it means the Medicare program will pay more," the authors said.

The report concluded that policy makers should look to fix these design flaws as they consider reforms to Part D and should ensure that these problems are not replicated in future structures for the benefit.

"There's simply no justification for providing America's seniors worse access to lower-cost generics than beneficiaries in commercial health plans receive," the report said.

"The system prevents seniors from getting the full value of their Part D benefit. Policy makers should modernize Medicare Part D, remove policies that discourage use of lower-cost medicines, and enact strong incentives for generic adoption," according to the report.

Policy makers should consider structural incentives to improve generic drug access, investigators say.

New Generics Are Less Available in Medicare Insurance Plans, Report Shows

Pfizer and BioNTech’s COVID-19 booster vaccines

 in Walgreens pharmacies nationwide, following the recent FDA Emergency Use Authorization and guidance from the CDC.

Those eligible for the booster doses include individuals 65 years of age and older; individuals 18 years of age and older who live in long-term care facilities; individuals with underlying medical conditions; and those who are at an increased risk for COVID-19 exposure and transmission because of their occupational or institutional setting. Patients should receive their booster dose at least 6 months after they completed their primary vaccine series.

“Walgreens has played a vital role in responding to COVID-19 as millions have turned to us as a convenient vaccination destination and critical point of community care,” said John Standley, president of Walgreens, in a press release. “In addition to providing booster shots, our pharmacy teams remain readily available to advise patients, address questions, and co-administer flu and COVID-19 primary vaccines to help protect our communities.”

In addition to these booster doses, individuals who are moderately to severely immunocompromised and who are already eligible for a third dose of the Pfizer or Moderna COVID-19 vaccine can also receive a booster dose 6 months after their primary series.

Co-administration of COVID-19 vaccines and flu vaccines is also available. The press release said that patients are encouraged to get their initial COVID-19 vaccination series or booster shot during the same visit as their flu shot to save time and protect against both viruses. Although COVID-19 booster shots are not approved for the wider public, the CDC urges nearly everyone to receive a flu shot to prevent flu-related hospitalizations and death.

Walgreens Announces Pfizer COVID-19 Vaccine Booster Shots Now Available Nationwide for Eligible Individuals. Walgreens; September 24, 2021. Accessed September 24, 2021. 

https://news.walgreens.com/press-center/walgreens-announces-pfizer-covid-19-vaccine-booster-shots-now-available-nationwide-for-eligible-individuals.htm

Co-administration of COVID-19 vaccines and flu vaccines is also available.

Walgreens Announces Availability of Pfizer COVID-19 Booster Vaccines for Eligible Patients

A new imaging technique using prostate-specific membrane antigen (PSMA)-targeted positron emission tomography (PET) imaging could help locate prostate cancer tumors for more precise treatment, according to a press release.

In addition to identifying cancer lesions in the prostate, the technique could help track cancer that has spread to other parts of the body, which is often missed by current standard-of-care imaging techniques. The tool utilizes PET scanning to identify a radioactive tracer that is highly effective in finding prostate cancer lesions throughout the body, enabling better visualization and more selective treatment.

Researchers at the University of California, Los Angeles and University of California, San Francisco investigated the technique, and according to the press release, their resulting paper led to an FDA approval for the imaging technology. The paper examined the use of the technology for primary staging of prostate cancer before any initial therapy is done.

When patients are diagnosed with prostate cancer and have some pathologic features suggesting risk of metastasis in the lymph nodes or bones, the press release said physicians should know whether the cancer has spread outside of the prostate. PSMA PET/CT scanning is a whole-body imaging modality with high accuracy rates for locating these potential metastases and is the best currently known modality for whole-body prostate cancer staging, according to the paper authors.

In the study, the scan was used in patients at initial staging prior to surgery. The investigators’ primary goal was to evaluate the performance of the scan to detect pelvic lymph node metastasis. The pelvic lymph nodes are commonly the first site of metastasis outside of the prostate, before the cancer has spread to the bone or other organs.

Importantly, the researchers said the only true way to verify whether the cancer is in the pelvic lymph node is to analyze the nodes following surgical removal. They evaluated the ability of the scan to detect some pelvic lymph nodes and identify them as cancerous, compared with the reference gold standard of histopathology analysis.

At the end of the study, only 277 of the 764 patients who received the scan underwent surgery. Approximately 64% of participants underwent another treatment instead of surgery because their scans showed some disease outside of the prostate and the surgeon felt that surgery was no longer ideal. Other treatment options included radiation and hormonal treatments, according to the press release.

Regarding the main goal of the study, the researchers found that the scan had 40% sensitivity, meaning it could detect pelvic lymph node disease in 40% of the patients who actually have pelvic lymph node disease. In 60% of patients, the lesions were still too small to be detected, although the researchers said this is better than any other imaging technique currently available. Furthermore, they noted that the sensitivity would have been much higher if all 764 patients underwent surgery, because most of the patients who did not undergo surgery had large lesions detected by the scan but were not included in the analysis.

In addition to these important findings, the researchers also found very high specificity of the test. When patients did not have pelvic lymph node metastasis, the scan was correctly negative in 95% of cases. Based on this, the investigators said that when the scan finds a positive lesion it is very likely prostate cancer.

According to the press release, the PSMA PET scan is now part of the National Comprehensive Cancer Network (NCCN) guidelines beginning in 2022. The NCCN panel wrote that they “do not feel that conventional imaging is a necessary prerequisite to PSMA-PET. They added that “PSMA-PET can serve as an equally effective, if not more effective, front-line imaging tool for these patients,” according to the press release.

UCLA Jonsson Comprehensive Cancer Center. Q&A: How a new imaging tool helps to better stage men with prostate cancer. September 16, 2021. Accessed September 23, 2021. 

https://cancer.ucla.edu/Home/Components/News/News/1740/1631

The prostate-specific membrane antigen-targeted positron emission tomography imaging technique will be part of the National Comprehensive Cancer Network guidelines beginning in 2022.